On October 6, 2020 Elorac Biotherapeutics, Inc., a biopharmaceutical company focused on developing innovative, best-in-class, proprietary drugs, reported it has amended its current clinical trial evaluating the safety and efficacy of topically applied naloxone hydrochloride lotion, 0.5%, for the treatment of moderate or severe pruritus in patients with the mycosis fungoides or Sézary Syndrome forms of Cutaneous T-cell Lymphoma (CTCL) (Press release, Elorac, OCT 6, 2020, View Source [SID1234568165]). The original double-blind, cross-over, multi-center Phase 3 study has been amended to provide access to naloxone lotion to all enrolled subjects for an additional six months after study completion.

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Scott B. Phillips, MD, Elorac’s Sr. V.P. Scientific Affairs, who earlier this year presented an update on this trial at the 4th World Congress of Cutaneous Lymphomas in Barcelona, hosted an industry breakout room on September 12, 2020 as part of the Cutaneous Lymphoma Foundation’s patient conference.

"The availability of naloxone lotion within the current Phase 3 study offers potential relief to people living with cutaneous T-cell lymphoma whose quality of life is significantly impaired by chronic, unrelenting itching," said Susan Thornton, CEO, Cutaneous Lymphoma Foundation.

"The availability of a topical medication to effectively treat the pruritus of mycosis fungoides and Sezary syndrome will be welcomed by our patients whose itch, in many cases, is inadequately treated by currently available medications, and not only impacts their quality of life but can increase their morbidity and mortality due to infection," adds Lucia Seminario-Vidal, MD, PhD, Associate Director, Clinical Research Unit, Dermatology and Cutaneous Surgery, University of South Florida and Co-director of the Multi-disciplinary Cutaneous Lymphoma Clinic, Moffitt Cancer Center.

Elorac received Orphan Drug Designation for naloxone hydrochloride lotion from both FDA and the European Medicines Agency. Elorac also has Fast Track designation from FDA for this novel investigational new drug. Fast Track designation provides for earlier and more frequent interaction with FDA during a drug’s development and eligibility for receiving priority review and accelerated approval from FDA. Elorac holds worldwide marketing rights to naloxone lotion.

About Naloxone

Naloxone is an opiate antagonist with no agonist activity. Intravenous, subcutaneous, and intranasal formulations of naloxone are used to treat opiate overdoses, and naloxone is used orally in combination with buprenorphine to treat opiate dependence. Naloxone lotion is an investigational new drug designed to relieve pruritus associated with CTCL and is not approved for marketing in the United States.

About Cutaneous T-Cell Lymphoma (CTCL)

CTCL affects approximately 30,000 patients in the United States, with an estimated 3,000 new cases diagnosed each year. During the course of this disease most patients will experience chronic intractable pruritus unresponsive to standard antipruritic agents (e.g., antihistamines and topical corticosteroids). In addition to a very detrimental impact on quality of life, chronic intractable pruritus has been associated with an increase in the mortality rate for individuals with CTCL. There are currently no approved therapeutic treatment options for pruritus associated with CTCL.

On October 6, 2020 A2 Biotherapeutics (www.a2bio.com), a biotechnology company developing innovative cell therapies for solid tumor cancer patients, reported the closing of its $71.5M Series B financing. Proceeds will fund the advancement of its Tmod (T-cell module) platform and the clinical development and in-house manufacturing of its three near-term product candidates (Press release, A2 Biotherapeutics, OCT 6, 2020, View Source [SID1234568164]). Tmod-engineered T cells uniquely combine a potent activating mechanism to kill tumor cells with a blocking mechanism that protects normal cells from harm by exploiting the loss of genetic material in tumors. "We are the first company to create robust engineered T cells that can integrate two signals in this way, harnessing the awesome power of immune cells to attack tumors that have lost specific genes, while sparing normal cells whose genomes are intact," said Scott Foraker, president and chief executive officer of A2.

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The Tmod design and proof-of-concept work was published last Friday in Molecular Immunology (Hamburger et al., 2020). This paper describes the Tmod dual-targeting system and demonstrates its abilities to recognize and selectively kill tumor cells that have lost expression of defined target molecules.

Tmod provides a practical solution to two major problems of cancer research: distinguishing tumor vs. normal cells and accessing new cancer targets. A2 plans to use Tmod to exploit the large, untapped source of targets that are lost in tumors by genetic deletion, thereby opening up new cancer targets that were previously unaddressable and making existing targets safer and more effective. The Tmod platform can produce many products using different activator/blocker combinations. By enabling these combinations, the Tmod platform has the potential to treat nearly all solid tumor cancers, the cause of approximately a half-million deaths/year in the U.S. alone.

A2 has an experienced team of former Amgen and Kite Pharma drug and cell therapy developers to fulfill the vast potential of these therapies. Alexander Kamb, co-founder and chief scientific officer of A2, said: "We have first-rate discovery scientists who can deliver on pioneering cancer programs and technology, as well as experienced development and manufacturing staff who enable us to innovate on both sides of the cell-therapy product—the Tmod target/receptor system and the T cells."

A2 Biotherapeutics, established in 2018, is a fully integrated discovery, development and manufacturing organization with more than 40 staff located in Agoura Hills, California. The company has raised $136M since inception. The company plans to use the Series B funds to support operations, including:

Development of three near-term product candidates, the first of which is slated to begin clinical testing in 2022
Extension of the Tmod platform to produce additional product candidates
Operation of A2’s completed, in-house cell-therapy manufacturing facility
Investors in the Series B include The Column Group, Vida Ventures, Samsara BioCapital, Nextech Invest, Casdin Capital, Euclidean Capital, UC Investments (Office of the Chief Investment Officer of the Regents) and Hartford HealthCare Endowment.

On October 6, 2020 Maze Therapeutics, a company focused on translating genetic insights into new medicines, reported that Sarah Noonberg, M.D., Ph.D., has been appointed as the company’s chief medical officer (Press release, Maze Therapeutics, OCT 6, 2020, View Source [SID1234568163]). Dr. Noonberg brings significant industry and clinical experience to Maze as the company works to advance its pipeline.

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"Sarah is an exceptional leader who has helped guide multiple companies through various stages of clinical development and commercialization, and we are thrilled to welcome her to the Maze team," said Jason Coloma, Ph.D., chief executive officer of Maze. "We have been working hard to build an exciting pipeline of potential therapeutics based on targets generated through our platform, and Sarah’s broad experience leading programs from translational research through commercialization for both rare and common diseases will be instrumental. As we take important steps toward entering the clinic and defining our development strategies, we could not be more excited to have her on board and for the future of Maze."

Dr. Noonberg is a board-certified physician-scientist with broad expertise and a proven track record in global clinical development and corporate strategy. Dr. Noonberg joins Maze from prior roles as chief medical officer of Nohla Therapeutics and Prothena Biosciences, and earlier, head of global clinical development at BioMarin Pharmaceutical, Inc., where she advanced a broad portfolio including cerliponase alfa (Brineura) for CLN2 disease, pegvaliase (Palynziq) for phenylketonuria, and gene therapy for hemophilia A. Prior to BioMarin, she was senior vice president at Medivation leading translational and early development activities as well as late-stage development of enzalutamide (XTANDI) for advanced prostate cancer. Dr. Noonberg currently serves on the board of directors of Protagonist Therapeutics and Neoleukin Therapeutics. She holds an M.D. from the University of California, San Francisco, a Ph.D. in bioengineering from the University of California, Berkley, and a B.S. in engineering science from Dartmouth College.

"Maze has assembled all of the key ingredients to produce a diverse pipeline of meaningful therapeutics that could significantly impact the treatment of patients with a broad range of diseases," said Dr. Noonberg. "I have been impressed by the company’s approach of leveraging human genetics insights and functional genomics to build an exceptional platform. I look forward to helping lead Maze’s programs through development, and ultimately, bringing them to patients in need."

On October 6, 2020 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported that its data will be featured in oral presentations during the 2020 American Society for Dermatologic Surgery (ASDS) Virtual Annual Meeting, October 9-11, 2020 (Press release, Castle Biosciences, OCT 6, 2020, View Source [SID1234568162]).

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Presentation details are as follows:

Title: Clinical validation and incorporation of a prognostic 40-gene expression profile test into clinicopathological risk assessment for cutaneous squamous cell carcinoma (cSCC)
Session: Skin Cancer & Reconstruction
Presenter: Sherrif Ibrahim M.D., Ph.D., associate professor, University of Rochester Medical Center
Date: Friday, October 9, 2020
Time: 6:24 p.m. – 6:27 p.m. ET

Title: Cutaneous melanoma prognostic model combining 31-gene expression profile and sentinel lymph node biopsy
Session: Skin Cancer & Reconstruction
Presenter: Aaron Farberg, M.D., Baylor University Medical Center, Dallas, Texas
Date: Friday, October 9, 2020
Time: 6:30 p.m. – 6:33 p.m. ET

The virtual presentations will be available to meeting registrants for 30 days following the meeting.

About DecisionDx-Melanoma

DecisionDx-Melanoma is a gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous melanoma metastasis or recurrence, as well as sentinel lymph node positivity, independent of traditional staging factors, and has been studied in more than 5,700 patient samples. Using tissue from the primary melanoma, the test measures the expression of 31 genes. The test has been validated in four archival risk of recurrence studies of 901 patients and six prospective risk of recurrence studies including more than 1,600 patients. Prediction of the likelihood of sentinel lymph node positivity has also been validated in two prospective multicenter studies that included more than 3,000 patients. Impact on patient management plans for one of every two patients tested has been demonstrated in four multicenter and single-center studies including more than 560 patients. The consistent performance and accuracy demonstrated in these studies provides confidence in disease management plans that incorporate DecisionDx-Melanoma test results. Through June 30, 2020, DecisionDx-Melanoma has been ordered more than 59,900 times for use in patients with cutaneous melanoma.

About DecisionDx-SCC

DecisionDx-SCC is a 40-gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of squamous cell carcinoma metastasis for patients with one or more risk factors. The test result, in which patients are stratified into a Class 1, 2A or 2B risk category, predicts individual metastatic risk to inform risk-appropriate management.

Peer-reviewed publications have demonstrated that DecisionDx-SCC is an independent predictor of metastatic risk and that integrating DecisionDx-SCC with current prognostic methods can add positive predictive value to clinician decisions regarding staging and management.

More information about the test and disease can be found at www.mySCCskincancer.com.

On October 6, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious blood diseases, reported that the Phase 3 study of omidubicel, an investigational advanced cell therapy in development as a potential life-saving treatment option for patients in need of bone marrow transplant, met all three of its secondary endpoints (Press release, Gamida Cell, OCT 6, 2020, View Source [SID1234568161]). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has the potential to be the first FDA-approved engineered bone marrow transplant graft.

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The international, multi-center, randomized Phase 3 study was designed to evaluate the safety and efficacy of omidubicel in patients with hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. In May, Gamida Cell reported that omidubicel achieved its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in recovery from a bone marrow transplant. The prespecified secondary endpoints of the study, analyzed in all randomized patients (intent-to-treat), were the proportion of patients who achieved platelet engraftment by day 42, the proportion of patients with Grade 2 or Grade 3 bacterial or invasive fungal infections in the first 100 days following transplant, and the number of days alive and out of the hospital in the first 100 days following transplant. All three secondary endpoints demonstrated a statistically significant improvement among patients who received omidubicel compared to the comparator group. The company anticipates reporting the full data set at a medical meeting in the fourth quarter of 2020.

"These data, obtained in a global, randomized, multi-institutional setting could represent an important step forward in the field. In addition to more rapid platelet engraftment, a key step toward recovery, reducing infections and hospitalizations are considered meaningful patient outcomes and have the potential to provide substantial value for patients, their families and the healthcare system," said Mitchell Horwitz, M.D., principal investigator and professor of medicine at the Duke Cancer Institute. "The totality of these data strengthen my belief that omidubicel has the potential to be a graft source for any patient who does not have access to a matched related donor and could help make stem cell transplantation more accessible and more successful for patients with lethal blood cancers."

"These additional data reinforce the potential of omidubicel and move us another step closer toward bringing potentially curative therapies to patients. We look forward to presenting data at a future medical meeting, and we are continuing our work to enable the submission of our biologics license application for omidubicel to the FDA on a rolling basis, both expected in the fourth quarter," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We deeply appreciate the patients who participated in this study, the incredible encouragement from their caregivers and the support we have received from investigators and their teams."

Despite the curative potential of bone marrow transplant, it is estimated that more than 40 percent of eligible patients in the United States do not receive a transplant for various reasons, including the lack of a matched donor.1 Even for patients who do receive a transplant, treatment is not always effective and can lead to serious complications that can dramatically affect their quality of life.2 Omidubicel is intended to address the current limitations of bone marrow transplant by providing a therapeutic dose of stem cells while preserving the cells’ functional therapeutic characteristics.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.3,4 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.